Patients with beta-thalassemia major (also known as Cooley's anemia) have a reduction or absence of beta protein in their hemoglobin, resulting in severe anemia, which requires regular blood ...

A domestically developed gene-editing therapy has successfully treated patients with beta-thalassemia, allowing them to live ...

Please provide your email address to receive an email when new articles are posted on . Patients with sickle cell disease and beta-thalassemia reported substantial improvements in quality of life ...

Novo Nordisk (NYSE:NVO) is advancing etavopivat through late stage clinical trials as a potential therapy for thalassemia. The treatment is under ongoing regulatory review, focused on both beta and ...

An essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires life-long blood transfusions for survival, non-transfusion-dependent ...

If Editas Medicine’s CRISPR gene-editing therapy for rare blood diseases reaches the market, it will be in the hands of a different company. The biotech on Tuesday announced plans to partner or ...

Aqvesme is expected to be available in late January 2026. The Food and Drug Administration (FDA) has approved Aqvesme™ (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia ...

Please provide your email address to receive an email when new articles are posted on . Children with beta thalassemia achieved transfusion independence after being treated with exa-cel. Children with ...

Your blood carries oxygen to all of the cells in your body. When there are problems with red blood cells, you may feel weak and tired and may have to take medicines or receive blood transfusions to ...

Good day, and welcome to Agios Pharmaceuticals' Investor Conference Call and Webcast. [Operator Instructions] Please be advised that this call is being recorded at Agios' request. I would now like to ...

In the beta thalassemia study, Dr. de la Fuente and his colleagues used two quality of life scales to evaluate outcomes in adult patients and two quality of life scales for adolescent patients. Unlike ...